There are other children born with disease and disability, like cystic fibrosis or juvenile diabetes.
Mutations in introns give rise to cystic fibrosis, breast cancer or forms of anemia.
The 26-year-old rock 'n' roller suffers from cystic fibrosis, which fills her lungs with bacteria-infested mucus.
The money raised has been split between Royal Manchester Children's Hospital and the Cystic Fibrosis Trust.
Nonsense mutations also cause 10% of cystic fibrosis cases, and the PTC drug shows promise there.
Cystic fibrosis is a life-threatening disorder that causes severe damage to the lungs and digestive system.
An inherited condition, cystic fibrosis affects the cells that produce mucus, sweat and digestive juices.
But in cystic fibrosis, a defective gene causes the secretions to become thick and sticky.
Cystic fibrosis is a serious genetic disorder which damages the lungs and digestive system.
The Cystic Fibrosis Trust said not funding the drug in Scotland would cause "dismay and heartache".
BBC: Call for cystic fibrosis drug to be available in Scotland
The Sun has defended its story revealing Mr Brown's youngest son Fraser had cystic fibrosis.
She's been in a Philadelphia hospital for months due to cystic fibrosis that she's had since birth.
The idea is this: Kalydeco works by improving the function of the Cystic Fibrosis Transmembrane Regulator protein.
FORBES: The Best Argument That Vertex's CF Drug Combination Works
It has worked phenomenally for organizations like the Cystic Fibrosis Foundation and the Multiple Myeloma Research Foundation.
The trust said funding of Kalydeco should not impact on the wider funding of cystic fibrosis care.
She started pilot programs in cystic fibrosis and bronchiolitis to see if doctors would follow stricter medical guidelines.
Cystic fibrosis is a symbol of how difficult it is to move from genetic discoveries to new medicines.
TOBI, a version of the antibiotic tobramycin, designed to treat fatal lung infections in patients with cystic fibrosis.
If these results continue to hold, we could potentially see an expedited path for less represented cystic fibrosis genotypes.
FORBES: Vertex Scores A Big Win With Its Cystic Fibrosis Experiment
The Cystic Fibrosis Trust had warned patients in Wales could have been faced with a second class health service.
In 2005 doctors in Australia noticed that surfers with cystic fibrosis had a lot less muck in their lungs.
It is often related to a certain cystic fibrosis trait which Frank inherited.
The Sun said a story it ran about Mr Brown's baby son having cystic fibrosis had been sourced legitimately.
BBC: Brown in ferocious Commons attack on News International
The average life expectancy for a cystic fibrosis patient is only 30 years.
The other 28, 800 or so people with cystic fibrosis are out of luck.
FORBES: The Best Argument That Vertex's CF Drug Combination Works
Some folks with cystic fibrosis, high serum triglycerides and other familial syndromes are at increased risk of acute pancreatitis.
Gordon Brown spoke with evident anguish and anger about The Sun's revelation that his youngest child had cystic fibrosis.
Leaving aside the cystic fibrosis case, he says genetic testing benefits from incentives for innovation by luring in investors.
If a new combination of Kalydeco and another cystic fibrosis pill proves effective, it would likely become a blockbuster.
The result is a big win for developer Vertex Pharmaceuticals and its partner, the Cystic Fibrosis Foundation, a patient group.
应用推荐